中国血友病B生物基因治疗研究状况

The bio-therapy and gene-therapy of hemophilia B in China

血友病B为一种严格的X连锁隐性遗传病,由于其单纯的分子遗传基础和明确的临床表现,容易观察疾病治疗的效果;hFⅨ较小的基因规模可以适用于几乎所有的常用基因载体系统.在多种细胞均可以获得有效的表达使得基因治疗时可以有广泛的细胞选择范围,同时也为生物工程制备重组蛋白药物提供了便利.而且,血友病B有天然和基因操作形成的动物模型便于治疗的临床前实验研究.基于上述理由,血友病B一直是遗传病基因生物治疗和重组蛋白药物制备的重要模型.我国在20世纪90年代成功完成了基因治疗血友病B的临床试验,对推进我国的基因治疗起到重要作用.本文简述我国在血友病B的生物基因治疗方面的研究情况与发展方向,希望为基因治疗和重组蛋白药物研究人员提供参考.

Hemophilia B, defected of clot factor FIX, is a strict X chromosome linked recessive disease. Hemophilia B is a very good model for gene therapy due to its simple molecular basis, typical clinical characters, easily carried by different gene vectors. Functional FIX protein can be expressed in different cells that provided the possibility of producing recombinant protein drug. Moreover, the existing reliable animal model of hemophilia B makes the research of treatment more convenience. Chinese contributed to the gene therapy by succeeding in hemophilia B gene therapy during the early 1990s. Here, we briefly reviewed the advance of the bio-therapy and gene-therapy of hemophilia B in China.